abstract：:Despite improvements in drug and device therapy for heart failure, hospitalization rates and mortality have changed little in the past decade. Randomized clinical trials using gene transfer to improve function of the failing heart are the focus of this review. Four randomized clinical trials of gene transfer in heart ...

journal_title：Human gene therapy

authors： Penny WF,Hammond HK

更新日期：2017-05-01 00:00:00

abstract：:Deficiency of glycogen branching enzyme (GBE) causes glycogen storage disease type IV (GSD IV), which is characterized by the accumulation of a less branched, poorly soluble form of glycogen called polyglucosan (PG) in multiple tissues. This study evaluates the efficacy of gene therapy with an adeno-associated viral (...

journal_title：Human gene therapy

authors： Yi H,Zhang Q,Brooks ED,Yang C,Thurberg BL,Kishnani PS,Sun B

更新日期：2017-03-01 00:00:00

abstract：:In a model of growth-restricted sheep pregnancy, it was previously demonstrated that transient uterine artery VEGF overexpression can improve fetal growth. This approach was tested in guinea-pig pregnancies, where placental physiology is more similar to humans. Fetal growth restriction (FGR) was attained through peri-...

journal_title：Human gene therapy

authors： Swanson AM,Rossi CA,Ofir K,Mehta V,Boyd M,Barker H,Ledwozyw A,Vaughan O,Martin J,Zachary I,Sebire N,Peebles DM,David AL

更新日期：2016-12-01 00:00:00

abstract：:PhD-trained biomedical scientists are moving into an increasingly diverse variety of careers within the sciences. However, graduate and postdoctoral training programs have historically focused on academic career preparation, and have not sufficiently prepared trainees for transitioning into other scientific careers. A...

journal_title：Human gene therapy

authors： Fuhrmann CN

更新日期：2016-11-01 00:00:00

abstract：:Adeno-associated virus (AAV) gene transfer is a promising treatment for genetic abnormalities. Optimal AAV vectors are showing success in clinical trials. Gene transfer to skeletal muscle and liver is being explored as a potential therapy for some conditions, that is, α1-antitrypsin (AAT) disorder and hemophilia B. Ex...

journal_title：Human gene therapy

authors： Carrig S,Bijjiga E,Wopat MJ,Martino AT

更新日期：2016-11-01 00:00:00

abstract：:Cell therapies are treatments in which stem or progenitor cells are stimulated to differentiate into specialized cells able to home to and repair damaged tissues. After their discovery, endothelial progenitor cells (EPCs) stimulated worldwide interest as possible vehicles to perform autologous cell therapy of tumors. ...

journal_title：Human gene therapy

authors： Laurenzana A,Margheri F,Chillà A,Biagioni A,Margheri G,Calorini L,Fibbi G,Del Rosso M

更新日期：2016-10-01 00:00:00

abstract：:Leukocyte adhesion deficiency type I (LAD-I) is a primary immunodeficiency caused by mutations in the ITGB2 gene and is characterized by recurrent and life-threatening bacterial infections. These mutations lead to defective or absent expression of β2 integrins on the leukocyte surface, compromising adhesion and extrav...

journal_title：Human gene therapy

authors： Leon-Rico D,Aldea M,Sanchez-Baltasar R,Mesa-Nuñez C,Record J,Burns SO,Santilli G,Thrasher AJ,Bueren JA,Almarza E

更新日期：2016-09-01 00:00:00

abstract：:Genome engineering has gone mainstream because of breakthroughs in defining and harnessing naturally occurring, customizable DNA recognition cursors (protein or RNA-guided). At present, most gene editing relies on these cursors to direct custom DNA endonucleases to a specific genomic sequence to induce a double-strand...

journal_title：Human gene therapy

authors： Martínez-Gálvez G,Ata H,Campbell JM,Ekker SC

更新日期：2016-06-01 00:00:00

abstract：:Isolated methylmalonic acidemia (MMA), a group of autosomal recessive inborn errors of metabolism, is most commonly caused by complete (mut(0)) or partial (mut(-)) deficiency of the enzyme methylmalonyl-CoA mutase (MUT). The severe metabolic instability and increased mortality experienced by many affected individuals,...

journal_title：Human gene therapy

authors： Harrington EA,Sloan JL,Manoli I,Chandler RJ,Schneider M,McGuire PJ,Calcedo R,Wilson JM,Venditti CP

更新日期：2016-05-01 00:00:00

abstract：:Cell-based therapy for muscular dystrophies was initiated in humans after promising results obtained in murine models. Early trials failed to show substantial clinical benefit, sending researchers back to the bench, which led to the discovery of many hurdles as well as many new venues to optimize this therapeutic stra...

journal_title：Human gene therapy

authors： Negroni E,Bigot A,Butler-Browne GS,Trollet C,Mouly V

更新日期：2016-02-01 00:00:00

abstract：:Adeno-associated viral (AAV) vectors containing cone-specific promoters have rescued cone photoreceptor function in mouse and dog models of achromatopsia, but cone-specific promoters have not been optimized for use in primates. Using AAV vectors administered by subretinal injection, we evaluated a series of promoters ...

journal_title：Human gene therapy

authors： Ye GJ,Budzynski E,Sonnentag P,Nork TM,Sheibani N,Gurel Z,Boye SL,Peterson JJ,Boye SE,Hauswirth WW,Chulay JD

更新日期：2016-01-01 00:00:00

abstract：:Current HIV-1 gene therapy approaches aim at stopping the viral life cycle at its earliest steps, such as entry or immediate postentry events. Among the most widely adopted strategies are CCR5 downregulation/knockout and the use of broadly neutralizing antibodies. However, the long-term efficacy and side effects are s...

journal_title：Human gene therapy

authors： Jung U,Urak K,Veillette M,Nepveu-Traversy MÉ,Pham QT,Hamel S,Rossi JJ,Berthoux L

更新日期：2015-10-01 00:00:00

abstract：:Urocortin-2 (UCn2) peptide infusion increases cardiac function in patients with heart failure, but chronic peptide infusion is cumbersome, is costly, and provides only short-term benefits. Gene transfer would circumvent these shortcomings. We previously showed that a single intravenous (IV) injection of AAV8.UCn2 incr...

journal_title：Human gene therapy

authors： Lai NC,Gao MH,Giamouridis D,Suarez J,Miyanohara A,Parikh J,Hightower S,Guo T,Dillmann W,Kim YC,Diaz-Juarez J,Hammond HK

更新日期：2015-06-01 00:00:00

abstract：:Mucopolysaccharidosis type II (MPS II) is a neuropathic lysosomal storage disorder caused by a deficiency of iduronate-2-sulfatase (IDS), which leads to the accumulation of glycosaminoglycans (GAGs). We demonstrated that biochemical alterations in the brains of MPS II mice are not corrected by bone marrow transplantat...

journal_title：Human gene therapy

authors： Wakabayashi T,Shimada Y,Akiyama K,Higuchi T,Fukuda T,Kobayashi H,Eto Y,Ida H,Ohashi T

更新日期：2015-06-01 00:00:00

abstract：:Recombinant vectors based on adeno-associated virus serotype 8 (AAV8) have been successfully used in the clinic and hold great promise for liver-directed gene therapy. Preexisting immunity against AAV8 or the development of antibodies against the therapeutic transgene product might negatively affect the outcomes of ge...

journal_title：Human gene therapy

authors： Ferla R,Claudiani P,Savarese M,Kozarsky K,Parini R,Scarpa M,Donati MA,Sorge G,Hopwood JJ,Parenti G,Fecarotta S,Nigro V,Sivri HS,Van Der Ploeg A,Andria G,Brunetti-Pierri N,Auricchio A

更新日期：2015-03-01 00:00:00

abstract：:Following in vivo recombinant adeno-associated virus (rAAV)-based gene transfer, adaptive immune responses specific to the vector or the transgene product have emerged as a potential roadblock to successful clinical translation. The occurrence of such responses depends on several parameters, including the route of vec...

journal_title：Human gene therapy

authors： Gernoux G,Guilbaud M,Dubreil L,Larcher T,Babarit C,Ledevin M,Jaulin N,Planel P,Moullier P,Adjali O

更新日期：2015-01-01 00:00:00

abstract：:Abstract Malignant gliomas (MGs) are highly vascularized, aggressive brain cancers carrying a dismal prognosis. Because of their high vascularity, anti-angiogenic therapy is a potential treatment option. Indeed, the anti-vascular endothelial growth factor (VEGF) antibody bevacizumab has demonstrated promising results ...

journal_title：Human gene therapy

authors： Samaranayake HD,Pikkarainen JT,Wirth T,Stedt H,Lesch HP,Dragneva G,Vuorio T,Määttä AM,Airenne K,Ylä-Herttuala S

更新日期：2014-11-01 00:00:00

abstract：:Extracellular vesicles (EVs) being released from two adjacent adeno-associated virus serotype 1 (AAV1)-producing 293T cells are shown by electron microscopy. We have shown that AAV vectors can associate with EVs and enter the media. Furthermore, we have recently reported that EV-associated AAV has robust gene delivery...

journal_title：Human gene therapy

authors： Fitzpatrick Z,György B,Skog J,Maguire CA

更新日期：2014-09-01 00:00:00

abstract：:The epithelial-mesenchymal transition (EMT) has been recognized to occur during embryonic development, fibrosis, and tumor metastasis. Nuclear factor (NF)-κB plays a central role in mediating the inflammation and wound-healing responses during liver fibrogenesis. However, the involvement of NF-κB during EMT in liver c...

journal_title：Human gene therapy

authors： Kim KH,Lee WR,Kang YN,Chang YC,Park KK

更新日期：2014-08-01 00:00:00

abstract：:The field of adenovirology is undergoing rapid change in response to increasing appreciation of the potential advantages of adenoviruses as the basis for new vaccines and as vectors for gene and cancer therapy. Substantial knowledge and understanding of adenoviruses at a molecular level has made their manipulation for...

journal_title：Human gene therapy

authors： Majhen D,Calderon H,Chandra N,Fajardo CA,Rajan A,Alemany R,Custers J

更新日期：2014-04-01 00:00:00

abstract：:Adenovirus type 5 (Ad5) is a commonly used vector for gene therapy, but its efficacy is limited by high seroprevalence and off-target hepatic and splenic sequestration. In order to circumvent these limitations, the use of vectors derived from rare species adenoviruses is appealing. The opportunity to retarget rare spe...

journal_title：Human gene therapy

authors： Coughlan L,Uusi-Kerttula H,Ma J,Degg BP,Parker AL,Baker AH

更新日期：2014-04-01 00:00:00

abstract：:Human adenoviruses are the most widely used vectors in gene medicine, with applications ranging from oncolytic therapies to vaccinations, but adenovirus vectors are not without side effects. In addition, natural adenoviruses pose severe risks for immunocompromised people, yet infections are usually mild and self-limit...

journal_title：Human gene therapy

authors： Hendrickx R,Stichling N,Koelen J,Kuryk L,Lipiec A,Greber UF

更新日期：2014-04-01 00:00:00

abstract：:Metabolic myopathies are a diverse group of rare diseases in which impaired breakdown of stored energy leads to profound muscle dysfunction ranging from exercise intolerance to severe muscle wasting. Metabolic myopathies are largely caused by functional deficiency of a single gene and are generally subcategorized into...

journal_title：Human gene therapy

authors： Mah CS,Soustek MS,Todd AG,McCall A,Smith BK,Corti M,Falk DJ,Byrne BJ

更新日期：2013-11-01 00:00:00

abstract：:Immune cells are involved in the pathogenesis of osteoarthritis (OA). CD4(+) T cells were activated during the onset of OA and induced macrophage inflammatory protein (MIP)-1γ expression and subsequent osteoclast formation. We evaluated the effects of local knockdown of MIP-1γ in a mouse OA model induced by anterior c...

journal_title：Human gene therapy

authors： Shen PC,Lu CS,Shiau AL,Lee CH,Jou IM,Hsieh JL

更新日期：2013-10-01 00:00:00

abstract：:Duchenne muscular dystrophy (DMD) typically occurs as a result of truncating mutations in the DMD gene that result in a lack of expression of the dystrophin protein in muscle fibers. Various therapies under development are directed toward restoring dystrophin expression at the subsarcolemmal membrane, including gene t...

journal_title：Human gene therapy

authors： Flanigan KM,Campbell K,Viollet L,Wang W,Gomez AM,Walker CM,Mendell JR

更新日期：2013-09-01 00:00:00

abstract：:Niemann-Pick type C (NP-C) disease is a neurodegenerative disorder characterized neuropathologically by ballooned neurons distended with lipid storage and widespread neuronal loss. Neural stem cells (NSC) derived from NP-C disease models have decreased ability for self-renewal and neuronal differentiation. Investigati...

journal_title：Human gene therapy

authors： Lee H,Kang JE,Lee JK,Bae JS,Jin HK

更新日期：2013-07-01 00:00:00

abstract：:Advances in cell and gene therapy are opening up new avenues for regenerative medicine. Because of their acquired pluripotency, human induced pluripotent stem cells (hiPSCs) are a promising source of autologous cells for regenerative medicine. They show unlimited self-renewal while retaining the ability, in principle,...

journal_title：Human gene therapy

authors： Garate Z,Davis BR,Quintana-Bustamante O,Segovia JC

更新日期：2013-06-01 00:00:00

abstract：:Recombinant adeno-associated viral (AAV) vectors of serotypes 6, 8, and 9 were characterized as tools for gene delivery to dopaminergic neurons in the substantia nigra for future gene therapeutic applications in Parkinson's disease. While vectors of all three serotypes transduced nigral dopaminergic neurons with equal...

journal_title：Human gene therapy

authors： Löw K,Aebischer P,Schneider BL

更新日期：2013-06-01 00:00:00

abstract：:Spinal muscular atrophy (SMA) is an autosomal recessive disease affecting ∼1 in 10,000 live births. The most striking component is the loss of α-motor neurons in the ventral horn of the spinal cord, resulting in progressive paralysis and eventually premature death. There is no current treatment paradigm other than sup...

journal_title：Human gene therapy

authors： Porensky PN,Burghes AH

更新日期：2013-05-01 00:00:00

abstract：:NKG2D ligands (NKG2DLs) are widely expressed on ovarian cancers to various degrees, making them attractive targets for immunotherapy. Here, we applied a chimeric antigen receptor (CAR) approach for the targeting of NKG2DLs expressed on human ovarian cancer cells and evaluated the impact of pharmacological upregulation...

journal_title：Human gene therapy

authors： Song DG,Ye Q,Santoro S,Fang C,Best A,Powell DJ Jr

更新日期：2013-03-01 00:00:00

abstract：:Here we show potent inhibition of HIV-1 replication in a human T cell line and primary human CD4(+) cells by expressing a single antiviral protein. Nullbasic is a mutant form of the HIV-1 Tat protein that was previously shown to strongly inhibit HIV-1 replication in nonhematopoietic cell lines by targeting three steps...

journal_title：Human gene therapy

authors： Apolloni A,Lin MH,Sivakumaran H,Li D,Kershaw MH,Harrich D

更新日期：2013-03-01 00:00:00

abstract：:Lentiviral vectors are efficient gene delivery vehicles for therapeutic and research applications. In contrast to oncoretroviral vectors, they are able to infect most nonproliferating cells. In the liver, induction of cell proliferation dramatically improved hepatocyte transduction using all types of retroviral vector...

journal_title：Human gene therapy

authors： Pichard V,Couton D,Desdouets C,Ferry N

更新日期：2013-02-01 00:00:00

abstract：:Stem cell mobilization to injured tissue contributes to neovascularization, resulting in regeneration after myocardial infarction (MI). We previously showed that direct cardiac injection of a recombinant lentivirus (LV) that engineers expression of membrane-bound stem cell factor (mSCF) improves outcomes immediately a...

journal_title：Human gene therapy

authors： Sun Z,Lee CJ,Mejia-Guerrero S,Zhang Y,Higuchi K,Li RK,Medin JA

更新日期：2012-12-01 00:00:00

abstract：:Genetically modified lymphoblastoid cell lines (LCL) have been shown to be an attractive alternative source of antigen-presenting cells for cancer vaccination in vitro. We tested their application in patients with pancreatic cancer in a phase I clinical trial. As a model tumor antigen, we selected the point-mutated (c...

journal_title：Human gene therapy

authors： Kubuschok B,Pfreundschuh M,Breit R,Hartmann F,Sester M,Gärtner B,König J,Murawski N,Held G,Zwick C,Neumann F

更新日期：2012-12-01 00:00:00

abstract：:Mucopolysaccharidosis type IIIA (MPSIIIA) is a rare lysosomal storage disorder caused by mutations in the sulfamidase gene. Accumulation of glycosaminoglycan (GAG) inside the lysosomes is associated with severe neurodegeneration as well as peripheral organ pathological changes leading to death of affected individuals ...

journal_title：Human gene therapy

authors： Ruzo A,Marcó S,García M,Villacampa P,Ribera A,Ayuso E,Maggioni L,Mingozzi F,Haurigot V,Bosch F

更新日期：2012-12-01 00:00:00

abstract：:Williams-Beuren syndrome (WBS) and supravalvular aortic stenosis (SVAS) are genetic syndromes marked by the propensity to develop severe vascular stenoses. Vascular lesions in both syndromes are caused by haploinsufficiency of the elastin gene. We used these distinct genetic syndromes as models to evaluate the feasibi...

journal_title：Human gene therapy

authors： Zhang P,Huang A,Morales-Ruiz M,Starcher BC,Huang Y,Sessa WC,Niklason LE,Giordano FJ

更新日期：2012-11-01 00:00:00

abstract：:Airway infiltration by eosinophils is a major characteristic of chronic asthma. CCL11 (eotaxin-1) is secreted by lung epithelial cells and functions as the major chemokine for eosinophil recruitment. Pseudotyped adeno-associated virus (AAV) 2/9, composed by the AAV2 rep and AAV9 cap genes, can efficiently target lung ...

journal_title：Human gene therapy

authors： Wu CJ,Huang WC,Chen LC,Shen CR,Kuo ML

更新日期：2012-11-01 00:00:00

abstract：:Skeletal muscle represents an attractive target tissue for adenoviral gene therapy to treat muscle disorders and as a production platform for systemic expression of therapeutic proteins. However, adenovirus serotype 5 vectors do not efficiently transduce adult muscle tissue. Here we evaluated whether capsid modificati...

journal_title：Human gene therapy

authors： Guse K,Suzuki M,Sule G,Bertin TK,Tyynismaa H,Ahola-Erkkilä S,Palmer D,Suomalainen A,Ng P,Cerullo V,Hemminki A,Lee B

更新日期：2012-10-01 00:00:00

abstract：:Newcastle disease virus (NDV) is a naturally oncolytic virus that has been shown to be safe and effective for cancer therapy. Tumor virotherapy using NDV emerged in the 1950s and has advanced more recently by the increased availability of reverse genetics technology. In this study, we constructed a reverse genetics sy...

journal_title：Human gene therapy

authors： Wei D,Sun N,Nan G,Wang Y,Liu HQ,Peeters B,Chen ZN,Bian H

更新日期：2012-07-01 00:00:00

abstract：:Engineered measles virus (MV) strains deriving from the vaccine lineage represent a promising oncolytic platform and are currently being tested in phase I trials. In this study, we have demonstrated that MV strains genetically engineered to express the human sodium iodide symporter (NIS) have significant antitumor act...

journal_title：Human gene therapy

authors： Opyrchal M,Allen C,Iankov I,Aderca I,Schroeder M,Sarkaria J,Galanis E

更新日期：2012-04-01 00:00:00